Clinical Trial Information for Patients

The following information is based on United States guidelines. Please check with your country’s requirements and regulations. To find Clinical Trials from The European Medical Agency go to

If you have questions regarding clinical trials, please contact us.

What are Clinical Trials?

In a clinical trial, participants receive specific interventions according to the research plan or protocol created by the investigators. These interventions may be medical products, such as drugs or devices; procedures; or changes to participants’ behavior, such as diet. Clinical trials may compare a new medical approach to a standard one that is already available to a placebo that contains no active ingredients, or to no intervention. Some clinical trials compare interventions that are already available to each other. When a new product or approach is being studied, it is not usually known whether it will be helpful, harmful, or no different than available alternatives (including no intervention). The investigators try to determine the safety and efficacy of the intervention by measuring certain outcomes in the participants. For example, investigators may give a drug or treatment to participants who have high blood pressure to see whether their blood pressure decreases.


Open-label Study to Determine the Maximum Tolerated Dose of DSG3-CAART in Mucosal-dominant PV Patients (mPV): More information about this trial


A Phase 2/3 Study of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid (BALLAD): More information about this trial

A Study to Evaluate the Efficacy and Safety of Dupilumab in Adult Patients With Bullous Pemphigoid (LIBERTY-BP): More information about this trial

A Study to Investigate the Use of Benralizumab in Patients With Bullous Pemphigoid (FJORD): More information about this trial

Telederm and Bullous Pemphigoid: More information about this trial

Find more pemphigus and pemphigoid clinical trials at

Clinical trial FAQs

The purpose of clinical trials is to collect data about the safety and efficacy of new drug and device development. There are several steps and stages of approval in the clinical trials process a drug or device must go through before it can be sold in the consumer market. If it fails any of the stages, it may not be approved. Since there are no devices used for P/P, this page will focus on treatments.

Drug testing begins with extensive laboratory research that can last years. During this time, experiments in animals and human cells will occur. If this initial laboratory research is successful, researchers send the data to the United States Food and Drug Administration (FDA) for approval to continue research and testing in humans. If approved, human testing of experimental drugs begins. This testing is usually done in five phases (Phase 0-IV) with each considered a separate trial. After each phase is completed, investigators must submit their data back to the FDA for approval before continuing to the next phase.

Human Clinical Trial Phases

Phase 0 exploratory studies involve very limited human exposure to the drug, with no therapeutic or diagnostic goals (for example, screening studies, microdose studies).

Phase I studies assess the safety of a drug and is the initial phase of testing. This phase can take several months to complete, usually including 20-100 healthy volunteers who are typically paid for their participation. This phase determines the effects of the drug on humans including how it is absorbed, metabolized, and excreted. This phase also investigates any side effects that occur as dosage levels are increased.

Phase II studies test the efficacy of a drug. This second phase of testing can last from several months to two years. It can involve several hundred patients. Most phase II studies are randomized trials where one group of patients receives the experimental drug, while a second “control” group receives a standard treatment, or placebo. These studies are “blinded” meaning neither the patients nor the researchers know who has received the experimental drug. This allows investigators to provide the pharmaceutical company and the FDA with comparative information about the relative safety and effectiveness of the new drug.

Phase III studies involve randomized and blind testing in several hundred to several thousand patients. This large-scale testing can last several years and provides the pharmaceutical company and the FDA with a thorough understanding of the effectiveness of the drug, its benefits, and the range of possible adverse reactions. Once Phase III is complete, a pharmaceutical company can request FDA approval for marketing the drug.

Phase IV studies (Post Marketing Surveillance Trials) are conducted after a drug has been approved for consumer sale. Pharmaceutical companies have several objectives at this stage: (1) to compare a drug with other drugs already in the market; (2) to monitor a drug’s long-term effectiveness and impact on a patient’s quality of life; and (3) to determine the cost-effectiveness of a drug therapy relative to other traditional and new therapies. Phase IV studies can result in a drug being taken off the market or restrictions of use could be placed on the product depending on the findings in the study.

Participating in a Clinical Trial

For detailed information and an FAQ about participating in clinical trials, please visit Should I Volunteer? And how?

Funding Clinical Trials

Funding for clinical research comes from the federal government (e.g., National Institutes of Health, Department of Defense, Department of Veteran’s Affairs), private industry (e.g., pharmaceutical and biotech companies), medical institutions, and foundations.

Participating in a clinical trial can be a difficult decision to make. Is it right for me? Would I qualify? What if I get worse? Can I quit? These are very real concerns and you should have those answers before deciding.

What is a clinical trial?

A clinical trial is a research study where volunteers receive investigational treatments under the supervision of a physician and research professionals. These treatments are developed by pharmaceutical and biotechnology companies who select qualified physicians, also known as investigators, to conduct clinical trials to determine the benefits of investigational drugs.

Who can participate in a clinical trial?

All clinical trials have guidelines about who can participate. Before joining a clinical trial, volunteers must qualify for the study. These factors are called “inclusion criteria” and the factors that disallow volunteers from participating are called “exclusion criteria.” These criteria can include age, gender, the type and stage of a disease, previous treatment history, and other medical conditions.

Some research studies seek participants with specific illnesses or conditions to be studied in a clinical trial (like PV or MMP opposed to anyone with pemphigus or pemphigoid). It is important to note that inclusion and exclusion criteria are used to identify appropriate participants, promote participants’ safety, and ensure that researchers learn the information they need.

How does a clinical trial work?

In a clinical trial, a volunteer is generally assigned a specific study group. Volunteers in one study group may receive an investigational treatment (the study drug) while other volunteers may receive a placebo (a treatment already available).

A placebo is an inactive product used to assess the experimental treatment’s effectiveness. The participant, physician, and research staff may not know which volunteer receives a placebo and which receives the active treatment. This is called a “blind study.” Not knowing which participants receive the active treatment lets physician and research staff objectively observe the volunteers during the study. Regardless of which treatment volunteers receive, the level of medical attention and care that each receives is the same.

What questions should be asked before choosing to participate?

Patients thinking about participating in a clinical trial should talk about it with their physicians and medical caregivers. Potential volunteers should know and understand the credentials and experience of the staff and the facility involved in conducting the study.

Questions to ask a physician or medical caregiver:

  • How long will the trial last?
  • Where is the trial being conducted?
  • What treatments will be used and how?
  • What is the main purpose of the trial?
  • How will patient safety be monitored?
  • Are there any risks involved?
  • What are the possible benefits?
  • What are the alternative treatments besides the one being tested in the trial?
  • Who is sponsoring the trial?
  • Do I have to pay for any part of the trial?
  • What happens if I am harmed by the trial?
  • Can I opt to remain on this treatment, even after termination of the trial?

What can volunteers expect if they participate?

Sometimes, participants receive a physical examination and their medical histories are reviewed once they are enrolled in the study. The volunteers’ health continues to be monitored during and after the trial. A detailed description of what is expected of volunteers will be outlined in consent forms and specific clinical trial information.

Sometimes it sounds like doctors and researchers speak a foreign language. “Phase II is a longitudinal, double-blind placebo…” From A to Z find we can help you find out what the Belmont Report is, what double-bind means, or what a pilot study is.

Get our Clinical Research Terms and Definitions Glossary here.